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Recent popular reports from Yimaike ★ Invitation Letter2021 CSGCT Gene and Cell Therapy Medical Summit will be held in Shanghai ★ In vitro lentiviral gene therapy presents positive test results, striving to free patients from the burden of chronic treatmentYimai Meng reveals November 2021 25th/eMedClub News/--Recently, REGENXBIO announced that the FDA has granted RGX-202 the title of "orphan drug", which is a potential one-time gene therapy for the treatment of Duchenne muscular dystrophy (Duchenne)
.
This also represents an important milestone in the development of RGX-202
.
Duchenne muscular dystrophy is a serious, progressive, and degenerative muscle disease caused by mutations in the DMD gene
.
The DMD gene encodes dystrophin, a cytoskeletal protein distributed on the plasma membrane side of skeletal muscle and cardiac muscle cell membranes, and acts as a cell scaffold to maintain muscle fiber integrity and anti-stretch function
.
Without functional dystrophin, muscles throughout the body will degenerate and become weak
.
People with Duchenne muscular dystrophy will develop progressive muscle weakness and eventually lose the ability to walk
.
RGX-202 aims to provide a new type of microdystrophin transgene that retains the key elements of dystrophin, including the extended coding region of the C-terminal (CT) domain found in naturally occurring dystrophin, And other fundamental improvements to genetic modification
.
The presence of the CT domain has been shown to recruit several key proteins to the muscle cell membrane, thereby improving the resistance of patients with Duchenne muscular dystrophy to contraction-induced muscle damage
.
RGX-202 is designed to use REGENXBIO's proprietary NAV AAV8 vector (a vector used in many clinical trials) and a well-characterized muscle targeting promoter (Spc5-12) to support the delivery and targeted expression of genes throughout the bone and myocardium
.
REGENXBIO, a company REGENXBIO, is a global leader in the development of gene therapies and AAV vectors, dedicated to improving lives through the potential of gene therapy
.
REGENXBIO’s NAV technology platform is a proprietary adeno-associated virus (AAV) gene delivery platform with exclusive rights to more than 100 new AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10
.
REGENXBIO’s gene therapy candidates all use AAV viral vectors from the NAV technology platform
.
The foundation of the NAV technology platform is to identify the next generation of AAV carriers, which can overcome the limitations of early AAV carriers (AAV1 to AAV6)
.
The main advantages of the NAV technology platform include: wide application in a variety of disease states; delivery of a variety of genetic material; simplified manufacturing compared to the previous generation of AAV vectors; compared with the previous generation of AAV vectors, it is unlikely to trigger an immune response; Compared with the first-generation AAV vector, gene expression has been improved, and it can achieve longer-lasting treatment with a smaller dose; it is derived from natural sources and can be modified for therapeutic purposes
.
REGENXBIO is developing gene therapy in the treatment of retinal, metabolic and neurodegenerative diseases
.
Currently, a number of candidate products have entered the clinical stage
.
▲ Development pipeline (picture source: REGENXBIO official website) ➤ RGX-314RGX-314 is under development as a potential one-time treatment for wet AMD, diabetic retinopathy and other chronic retinal diseases treated with anti-VEGF
.
It has now entered clinical phase 3 trials
.
Recommended reading: Nearly 1.
8 billion US dollars! AbbVie invests heavily in the field of ophthalmic gene therapyYi Mai Meng reveals that the market is still expanding, and at a glance treatment strategies for age-related macular degenerationYi Mai Hei Technology ➤ RGX-121RGX-121 is used for the treatment of type II mucopolysaccharidosis (MPS II) (also known as Hunter syndrome) product candidate
.
RGX-121 is designed to use AAV9 vector to deliver human iduronate-2-sulfatase (IDS) gene to the central nervous system (CNS)
.
➤ RGX-111RGX-111 is a product candidate for the treatment of type I mucopolysaccharidosis (MPS I)
.
RGX-111 is designed to use the AAV9 vector to deliver the human α-l-iodoketonease (IDUA) gene to the central nervous system (CNS)
.
➤ RGX-181RGX-181 is a product candidate for the treatment of neuronal meningeal fatty browning type 2 (CLN2 disease) in late infants, which is a form of Batten disease
.
RGX-181 is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene directly to the central nervous system (CNS)
.
➤ RGX-381 RGX-381 is a product candidate for the treatment of ocular manifestations of neuronal lipofuscinosis type 2 (CLN2 disease) in late infants
.
RGX-381 is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene directly to the retina
.
➤ RGX-202RGX-202 uses the AAV8 vector to deliver the gene encoding micro-dystrophin to muscle cells.
Micro-dystrophin is a shortened and functional form of dystrophin
.
Other RGX-202 features are designed to improve gene expression and reduce immunogenicity
.
At present, the research and development of RGX-202 is still in the pre-clinical development stage
.
REGENXBIO looks forward to bringing this one-time gene therapy to the clinic as soon as possible
.
Reference materials: 1.
https:// -treatment-of-duchenne-muscular-dystrophy-301429654.
html2.
https://
.
This also represents an important milestone in the development of RGX-202
.
Duchenne muscular dystrophy is a serious, progressive, and degenerative muscle disease caused by mutations in the DMD gene
.
The DMD gene encodes dystrophin, a cytoskeletal protein distributed on the plasma membrane side of skeletal muscle and cardiac muscle cell membranes, and acts as a cell scaffold to maintain muscle fiber integrity and anti-stretch function
.
Without functional dystrophin, muscles throughout the body will degenerate and become weak
.
People with Duchenne muscular dystrophy will develop progressive muscle weakness and eventually lose the ability to walk
.
RGX-202 aims to provide a new type of microdystrophin transgene that retains the key elements of dystrophin, including the extended coding region of the C-terminal (CT) domain found in naturally occurring dystrophin, And other fundamental improvements to genetic modification
.
The presence of the CT domain has been shown to recruit several key proteins to the muscle cell membrane, thereby improving the resistance of patients with Duchenne muscular dystrophy to contraction-induced muscle damage
.
RGX-202 is designed to use REGENXBIO's proprietary NAV AAV8 vector (a vector used in many clinical trials) and a well-characterized muscle targeting promoter (Spc5-12) to support the delivery and targeted expression of genes throughout the bone and myocardium
.
REGENXBIO, a company REGENXBIO, is a global leader in the development of gene therapies and AAV vectors, dedicated to improving lives through the potential of gene therapy
.
REGENXBIO’s NAV technology platform is a proprietary adeno-associated virus (AAV) gene delivery platform with exclusive rights to more than 100 new AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10
.
REGENXBIO’s gene therapy candidates all use AAV viral vectors from the NAV technology platform
.
The foundation of the NAV technology platform is to identify the next generation of AAV carriers, which can overcome the limitations of early AAV carriers (AAV1 to AAV6)
.
The main advantages of the NAV technology platform include: wide application in a variety of disease states; delivery of a variety of genetic material; simplified manufacturing compared to the previous generation of AAV vectors; compared with the previous generation of AAV vectors, it is unlikely to trigger an immune response; Compared with the first-generation AAV vector, gene expression has been improved, and it can achieve longer-lasting treatment with a smaller dose; it is derived from natural sources and can be modified for therapeutic purposes
.
REGENXBIO is developing gene therapy in the treatment of retinal, metabolic and neurodegenerative diseases
.
Currently, a number of candidate products have entered the clinical stage
.
▲ Development pipeline (picture source: REGENXBIO official website) ➤ RGX-314RGX-314 is under development as a potential one-time treatment for wet AMD, diabetic retinopathy and other chronic retinal diseases treated with anti-VEGF
.
It has now entered clinical phase 3 trials
.
Recommended reading: Nearly 1.
8 billion US dollars! AbbVie invests heavily in the field of ophthalmic gene therapyYi Mai Meng reveals that the market is still expanding, and at a glance treatment strategies for age-related macular degenerationYi Mai Hei Technology ➤ RGX-121RGX-121 is used for the treatment of type II mucopolysaccharidosis (MPS II) (also known as Hunter syndrome) product candidate
.
RGX-121 is designed to use AAV9 vector to deliver human iduronate-2-sulfatase (IDS) gene to the central nervous system (CNS)
.
➤ RGX-111RGX-111 is a product candidate for the treatment of type I mucopolysaccharidosis (MPS I)
.
RGX-111 is designed to use the AAV9 vector to deliver the human α-l-iodoketonease (IDUA) gene to the central nervous system (CNS)
.
➤ RGX-181RGX-181 is a product candidate for the treatment of neuronal meningeal fatty browning type 2 (CLN2 disease) in late infants, which is a form of Batten disease
.
RGX-181 is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene directly to the central nervous system (CNS)
.
➤ RGX-381 RGX-381 is a product candidate for the treatment of ocular manifestations of neuronal lipofuscinosis type 2 (CLN2 disease) in late infants
.
RGX-381 is designed to use the AAV9 vector to deliver the tripeptidyl peptidase 1 (TPP1) gene directly to the retina
.
➤ RGX-202RGX-202 uses the AAV8 vector to deliver the gene encoding micro-dystrophin to muscle cells.
Micro-dystrophin is a shortened and functional form of dystrophin
.
Other RGX-202 features are designed to improve gene expression and reduce immunogenicity
.
At present, the research and development of RGX-202 is still in the pre-clinical development stage
.
REGENXBIO looks forward to bringing this one-time gene therapy to the clinic as soon as possible
.
Reference materials: 1.
https:// -treatment-of-duchenne-muscular-dystrophy-301429654.
html2.
https://
